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Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)

This study is currently recruiting participants.
Verified February 2013 by NKT Therapeutics
Sponsor:
Information provided by (Responsible Party):
NKT Therapeutics
ClinicalTrials.gov Identifier:
NCT01783691
First received: February 1, 2013
Last updated: February 13, 2013
Last verified: February 2013
History of Changes
  Purpose

The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.


Condition Intervention Phase
Sickle Cell Disease
 Drug: NKTT120
 Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single Ascending Dose Phase 1 Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biologic Activity of Intravenous NKTT120 in Adults With Stable Sickle Cell Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by NKT Therapeutics:

Primary Outcome Measures:
  • Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days ] [ Designated as safety issue: No ]
  • Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets [ Time Frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Pain scores as assessed by daily electronic diary [ Time Frame: Daily for 120 days ] [ Designated as safety issue: No ]
  • Quality of Life as assessed by two separate Quality of Life instruments [ Time Frame: 0, 30, 60, 90, 120 days ] [ Designated as safety issue: No ]
  • Lung function as measured by pulmonary function tests [ Time Frame: 0; 30 minutes; 30, 60, 90, 120 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 21
Study Start Date: February 2013
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NKTT120 Drug: NKTT120

  Eligibility

Ages Eligible for Study:   18 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 to 50 years
  • Subject has a confirmed diagnosis of HbSS or HbSβ0thal
  • Subject has stable SCD defined as not having acute VOC, ACS, or other major SCD associated event during the month prior to enrollment

Exclusion Criteria:

  • Subject had an SCD-related VOC or ACS that required hospitalization or treatment in acute care outpatient setting in the month prior to enrollment
  • Subject requires a program of prescheduled regularly administered packed red blood cell (pRBC) transfusions or Subject received a pRBC transfusion in the month prior to enrollment
  • Subject has evidence of latent or active tuberculosis
  • Subject has a major concurrent illness or medical condition
  • Subject is pregnant or nursing
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01783691

Contacts
Contact: Cheryl Eaton 7816840290 ceaton@nktrx.com

Locations
United States, Wisconsin
Blood Center of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Debora Nischik, RN CCRC     414-937-6457     debora.nischik@bcw.edu    
Sponsors and Collaborators
NKT Therapeutics
Investigators
Study Director: Rosemary Mazanet, MD NKT Therapeutics
  More Information

No publications provided

Responsible Party: NKT Therapeutics
ClinicalTrials.gov Identifier: NCT01783691     History of Changes
Other Study ID Numbers: 120-SCD1
Study First Received: February 1, 2013
Last Updated: February 13, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by NKT Therapeutics:
Sickle cell disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 19, 2013